The invention provides siRNA compositions that specifically downregulates expression of a variant of the PNPLA3 gene and methods of use thereof for treating a chronic liver disease or alcoholic liver disease (ALD).

Aspects of the invention include a polymer comprising: a plurality of repeating units, each repeating unit comprising a polymer backbone group directly or indirectly covalently linked to one or two side chain moieties; wherein: each polymer backbone group is independently a ROMP-polymerized monomer; each one of the one or two side chain moieties independently comprises a peptide moiety or a non-peptide therapeutic moiety; wherein the polymer comprises a plurality of peptide moieties; each polymer backbone group is covalently attached to at least one other polymer backbone group; 100% of the ROMP-polymerized monomers are each individually attached to the one or two side chain moieties; and at least one side chain moiety of the polymer comprises a non-peptide therapeutic moiety, one polymer-terminating group comprises a non-peptide therapeutic moiety, and/or each of both polymer-terminating groups comprises a non-peptide therapeutic moiety.

Aspects of the invention include a polymer comprising: a plurality of repeating units, each repeating unit comprising a polymer backbone group directly or indirectly covalently linked to one or two side chain moieties; wherein: each polymer backbone group is independently a ROMP-polymerized monomer; each one of the one or two side chain moieties independently comprises a peptide moiety or a non-peptide therapeutic moiety; wherein the polymer comprises a plurality of peptide moieties; each polymer backbone group is covalently attached to at least one other polymer backbone group; 100% of the ROMP-polymerized monomers are each individually attached to the one or two side chain moieties; and at least one side chain moiety of the polymer comprises a non-peptide therapeutic moiety, one polymer-terminating group comprises a non-peptide therapeutic moiety, and/or each of both polymer-terminating groups comprises a non-peptide therapeutic moiety.

Compositions and methods are provided according to aspects of the present disclosure for inhibition of a pathogenic virus. According to particular aspects, compositions and methods of the present disclosure include degradation of pathogenic viral nucleic acids by interferon-stimulated gene 20-kDa protein (ISG20) and/or a variant thereof, administered to a cell and/or subject to inhibit a pathogenic viral infection of the cell and/or a subject. Compositions and methods are provided according to aspects of the present disclosure wherein providing the ISG20 protein, or a variant thereof, comprises administering a therapeutically effective amount of the ISG20 protein and/or variant, to a subject having, or at risk of having, a viral infection.

Compositions and methods are provided according to aspects of the present disclosure for inhibition of a pathogenic virus. According to particular aspects, compositions and methods of the present disclosure include degradation of pathogenic viral nucleic acids by interferon-stimulated gene 20-kDa protein (ISG20) and/or a variant thereof, administered to a cell and/or subject to inhibit a pathogenic viral infection of the cell and/or a subject. Compositions and methods are provided according to aspects of the present disclosure wherein providing the ISG20 protein, or a variant thereof, comprises administering a therapeutically effective amount of the ISG20 protein and/or variant, to a subject having, or at risk of having, a viral infection.

A fatty acid-biocompatible polymer-photosensitizer conjugate is provided. The conjugate can kill GIP-secreting cells by generating active oxygen upon irradiation with light, and has the effect of increasing insulin, and thus can be effectively used for ameliorating and treating metabolic diseases such as obesity and diabetes.

The present invention relates to vaccines comprising at least one mRNA encoding at least one antigen for use in the treatment of a disease in newborns and/or infants, preferably exhibiting an age of not more than 2 years, preferably of not more than 1 year, more preferably of not more than 9 months or even 6 months, wherein the treatment comprises vaccination of the newborn or infant and eliciting an immune response in said newborn or infant. The present invention is furthermore directed to kits and kits of parts comprising such a vaccine and/or its components and to methods applying such a vaccine or kit.

The present invention relates to vaccines comprising at least one mRNA encoding at least one antigen for use in the treatment of a disease in newborns and/or infants, preferably exhibiting an age of not more than 2 years, preferably of not more than 1 year, more preferably of not more than 9 months or even 6 months, wherein the treatment comprises vaccination of the newborn or infant and eliciting an immune response in said newborn or infant. The present invention is furthermore directed to kits and kits of parts comprising such a vaccine and/or its components and to methods applying such a vaccine or kit.

An antibody includes a polyamidoamine (PAMAM) dendrimer and a first functional group. The polyamidoamine (PAMAM) dendrimer includes a plurality of branches and each of the branches has a phenylboronic acid (PBA) terminal group. The first functional group is bonded to at least one of the PBA terminal groups.

Provided herein are peptides comprising an amino acid sequence having at least about 85% sequence identity to RYRPRAPIIAVT (SEQ ID NO: 1). These cationic peptides inhibit PKM2 methylation and may be used in the treatment of breast cancer and other diseases or conditions in which PKM2 is overexpressed. Such PKM2 peptides may be delivered to cancer cells using pH sensitive unimolecular nanoparticles comprising anionic polymers.